Accelerating Drug Development

Duchenne UK is not only the largest funder of DMD research in the UK in the last 5 years. We are also committed to accelerating the pace of research, ensuring that we remove potential obstacles and roadblocks that lie in the path of drug development. It is vital that the research we fund has the potential to translate into clinical trials.

So we work with industry, engage with policy makers and regulators and also fund the doctors, nurses and physiotherapists to run clinical trials. For example we funded the Phase I study of VBP15 in the US. We then co-funded Dr Guglieri at Newcastle, who will be running the VBP15 trial in the UK. We proactively invest in every stage of the drug development timeline:

We support and encourage national and international collaboration to allow researchers and clinicians to collaborate and progress research. We are proud to support the Treat-NMD network and their bi-annual TACT meetings to review potential clinical trials for Duchenne Muscular Dystrophy.

Summit Therapeutics plc have announced positive 24-week interim data from PhaseOut DMD, their Phase 2 clinical trial of the utrophin modulator ezutromid. The data showed a significant reduction in muscle damage and an increase in utrophin in muscle biopsies.

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Our co-founders Alex Johnson and Emily Crossley met after their sons were diagnosed with Duchenne muscular dystrophy. They both set up charities, Alex with Joining Jack, Emily with the Duchenne Children’s Trust.

Duchenne Direct is a monthly Direct Debit from you to Duchenne UK, that is spent DIRECTLY on research.
100% of your donations will be ring-fenced and will go directly into our exciting research projects.
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